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Positive Results Announced for Aubagio in Phase III Study of People at High Risk for MS

April 26, 2013

Among 618 people at high risk of developing MS, significantly fewer people taking oral Aubagio® (teriflunomide, Genzyme, a Sanofi company) for two years had developed clinically definite MS than those taking placebo, Genzyme announced in a press release dated April 25, 2013. People entered into this study had experienced a first neurological episode and had brain MRI findings suggestive of MS (clinically isolated syndrome, CIS), but had not been diagnosed with definite MS. Further results of this study, also known as the TOPIC study, are expected to be presented at a forthcoming scientific meeting.

Background: CIS is a syndrome in which a person experiences a single neurological event suggestive of myelin damage, such as focal weakness, numbness, coordination problems, or decrease in vision in one eye, and also shows brain magnetic resonance imaging findings suggestive of MS. People with CIS may never develop definite MS, or it can be a prelude to being diagnosed with definite MS. The indication for several disease-modifying therapies for MS has been expanded by the U.S. Food and Drug Administration (FDA) to include people with CIS based on clinical trials showing benefits in delaying the risk of developing definite MS. (Read more). To date, there is no oral therapy that is approved for treating CIS.

Aubagio was approved by the FDA in September 2012 for people with relapsing forms of multiple sclerosis. Aubagio is an oral compound that inhibits the function of specific immune cells that have been implicated in MS. Read more about Aubagio .

The Study: Investigators worldwide recruited 618 people 18-55 years of age with CIS who showed two or more areas of damage on MRI scans and had experienced their neurological symptoms within 90 days prior to entering the study.

Participants were randomly assigned to receive 7 mg teriflunomide, 14 mg or placebo once daily for 108 weeks. The primary outcome measured was whether or not people developed clinically definite MS (as defined by the occurrence of a second clinical attack).

Reporting on the primary endpoint, the press release states that at two years, people taking Aubagio were significantly less likely to develop definite MS. The risk of conversion to definite MS was reduced by 43% among those taking 14 mg of Aubagio, and by 37% among those taking 7 mg Aubagio, compared to those taking placebo. The results of other endpoints are not included in the press release.

The most common adverse events reported in the Aubagio groups were liver enzyme elevations, nasal inflammation, headache, hair thinning, diarrhea and paresthesia (e.g., burning sensations, pins and needles, stabbing pains). There was one death due to suicide in the placebo arm. The rate of treatment discontinuation due to adverse events was 12.1% among those taking 7 mg Aubagio, 9.1 % in placebo arm, and 8.3% in those taking 14 mg Aubagio.

Next Steps: The press release notes that additional results of this trial will be presented at an upcoming medical meeting. A possible next step would be for the company to apply to the FDA and other regulatory agencies for expansion of Aubagio’s indication to include treatment of CIS.

About Multiple Sclerosis

Multiple sclerosis, an unpredictable, often disabling disease of the central nervous system, interrupts the flow of information within the brain, and between the brain and body. Symptoms range from numbness and tingling to blindness and paralysis. The progress, severity and specific symptoms of MS in any one person cannot yet be predicted, but advances in research and treatment are moving us closer to a world free of MS. Most people with MS are diagnosed between the ages of 20 and 50, with at least two to three times more women than men being diagnosed with the disease. MS affects more than 2.3 million people worldwide.